Carbaglu (Page 2 of 6)

3. DOSAGE FORMS AND STRENGTHS

Tablets for oral suspension: 200 mg of carglumic acid, white, elongated, functionally scored with 3 lines (for splitting into 4 equal portions) and printed “C” on one side.

4. CONTRAINDICATIONS

None

6. ADVERSE REACTIONS

6.1 Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Acute and Chronic Hyperammonemia due to NAGS DeficiencyIn a retrospective case series of 23 NAGS deficiency patients treated with CARBAGLU, 17 of the 23 patients reported an adverse reaction. The most common adverse reactions (occurring in ≥ 13% of patients) were vomiting, abdominal pain, pyrexia, tonsillitis, anemia, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, and headache.

Table 1 summarizes adverse reactions occurring in 2 or more patients treated with CARBAGLU.

Table 1: Adverse Reactions Reported in ≥ 2 Patients with NAGS deficiency Treated with CARBAGLU in the Retrospective Case Series
Adverse Reaction Number of Patients (N) (%)
Vomiting 6 (26)
Abdominal pain 4 (17)
Pyrexia 4 (17)
Tonsillitis 4 (17)
Anemia 3 (13)
Diarrhea 3 (13)
Ear infection 3 (13)
Infections 3 (13)
Nasopharyngitis 3 (13)
Hemoglobin decreased 3 (13)
Headache 3 (13)
Dysgeusia 2 (9)
Asthenia 2 (9)
Hyperhidrosis 2 (9)
Influenza 2 (9)
Pneumonia 2 (9)
Weight decreased 2 (9)
Anorexia 2 (9)
Somnolence 2 (9)
Rash 2 (9)

Acute Hyperammonemia due to PA and MMAIn a randomized, double-blind, placebo-controlled clinical trial, 24 patients (15 with PA and 9 with MMA) experienced a total of 90 hyperammonemic episodes which were randomized 1:1 to be treated with either CARBAGLU or placebo, each in addition to standard-of-care therapy, with randomization based on each hyperammonemic episode. The average patient age (SD) was 9.2 years (7.7) and 12 (50 %) were males.

CARBAGLU was administered at a dosage of 150 mg/kg/day for patients ≤15 kg or 3.3 g/m2 /day for patients >15 kg, divided into 2 doses, for a median duration of 4.14 days.

At least 1 adverse reaction was reported during the course of hyperammonemic episodes in 42% of hyperammonemic episodes. The most common adverse reactions (≥5%) during hyperammonemic episodes were neutropenia, anemia, vomiting, electrolyte imbalance, decreased appetite, hypoglycemia, lethargy/stupor, encephalopathy and pancreatitis/lipase increased.

Table 2 summarizes adverse reactions (≥2%) during hyperammonemic episodes in patients with PA or MMA treated with CARBAGLU or placebo.

Table 2: Adverse Reactions (≥ 2%) During Hyperammonemic Episodes in Patients with PA or MMA Treated with CARBAGLU or Placebo
Adverse Reaction Treatment Arm
CARBAGLU N=42 episodes PLACEBO N=48 episodes
N (%) N (%)
Neutropenia 6 (14) 4 (8)
Anemia 5 (12) 4 (8)
Vomiting 3 (7) 1 (2)
Electrolyte imbalance 3 (7) 2 (4)
Decreased appetite 2 (5) 1 (2)
Hypoglycemia 2 (5) 1 (2)
Lethargy/Stupor 2 (5) 1 (2)
Encephalopathy 2 (5) 0 (0)
Pancreatitis/Lipase increased 2 (5) 0 (0)
Cardiomyopathy 1 (2) 0 (0)
Alanine aminotransferase increased 1 (2) 0 (0)
Aspartate aminotransferase increased 1 (2) 0 (0)
Infusion site extravasation 1 (2) 0 (0)
White blood cell count increased 1 (2) 0 (0)
Behavior disorder 1 (2) 0 (0)
Sleep disorder 1 (2) 0 (0)
Apnea 1 (2) 0 (0)
Hyperventilation 1 (2) 0 (0)

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